26 October 2018

NACFC 2018 - 16 Things I Learned at NACF 2018

I just returned from being in Denver, Colorado for the 2018 North American Cystic Fibrosis Conference, an annual conference with more than 5,000 scientists, researchers, clinicians, parents and people with CF in attendance this year.

I wanted to do a "brain dump" blogpost so I won't forget all that I learned during my few days there (this post).  But I also wanted to reflect on some inner thoughts that emerged while I was there (next post).

For the first time ever, Brian came with me this year (we tried to steal away some time for a very rare weekend getaway for ourselves).  Brian was able to attend a few of the microbiology sessions and report back what he learned.

The most important point I should share (something have to remind myself each year) is that, in many ways, CF research is moving fast and, in other ways, CF research is slow.  Most of what we learn at NACFC each year, for the most part, isn't necessarily "groundbreaking" or rather, it doesn't often translate in to an immediate behavior change in CF Care.  But, taken in totality, it is helpful to learn to better understand the research trends and to get an idea of what is coming down the pipeline.

Here are the 16 things I learned at NACFC this year:

1.) Next year is gonna be a big year for cystic fibrosis!
Probably the biggest news was announced during the first Plenary.  We are likely less than one year away from the announcement of the "triple combo" - a Vertex drug (a combination of three drugs) that could benefit up to 90% of the CF Community (those who have 1 copy of the F508del).

We know that the "Triple Combo" drug is highly effective in people with a single copy of F508del. Phase II data results came out in February 2019.  Both VX-659 + Tezacaftor/Ivacaftor and VX-445 + Tezacaftor/Ivacaftor achieved similar results.

Data on the VX-659 trial should be available in late 2018.  Data on the VX-445 trial should be in the first quarter of 2019.  Vertex plans to submit approval no later than mid-2019.  

2.) Vertex isn't the only company advancing a triple combo drug.  

Proteostasis Therapeutics has multiple trials going on right now.  Galapogos does as well.  (Learn more about what is happening in this space by watching the NACFC 2018 Plenary 1 here: - starts about the 25 minute mark)

3.) CFTR Modulators are good.  But they aren't gonna make lung decline go away entirely.  

We know that lung decline is still an issue for those who are currently benefitting from CFTR modulators.  So, our community, even with modulators, needs to keep focused on keeping CF lungs healthy.

This slide, which shows evidence of Ivacaftor treatment and stopping Ivacaftor treatment, reminded me both how much I'm grateful for the CFTR modulators and how they are still not a cure.

This slide is a reminder that, CFTR modulators on the market or not, we still have a lot of reasons why we need to better understand respiratory infections and inflammation.

4.) The CFF is putting $100 million dollars towards an infection research initiative because we have so much more to learn about infection diagnosis, treatment and outcomes.

The CF Foundation recognizes that while CFTR modulators are coming down the pipeline, those for whom cannot benefit from this new drug combination have many years left to wait for their drug to come...and even then, there is some portion of the CF community who won't be able to benefit from these drugs just due to medication interference or some other reason.  Many people with CF are being plagued by lung infections and fungal infections.  That's why the CF Foundation has decided to invest $100 million to study these life-threatening infections affecting people with CF.  (Watch more about this initiative and the research that was presented during this Plenary here: - it starts about the 9 minute mark)

5.) Inflammation remains a major cause for lung damage.  There are new anti-inflammatory drugs in the pipeline.

There are new mucociliary clearance drugs also in the pipeline.

6.) The CF Foundation is putting resources towards using "the oldest new idea to improve CF Care": CoProduction (true partnership between the person with CF/families and the clinician/clinical team when making decisions in care)    

So many people enjoyed the last plenary of NACFC 2018 which was led by Maren Batalden.  Watch it here:

Maren talked about the general healthcare ecosystem, calling this slide a "map" of sorts.  Notice patients, professionals and the healthcare system overlap but all work towards health outcomes.

This was one of my favorite slides.  It demonstrates the relationship between the patient and the clinician.  Many times I've heard clinicians explain their interactions with patients as the first image ("communications").  Even at NACFC there was conversation about "educating" the patient and how the patient needed to be further "educated." But, the place where coproduction actually exists is within the last image of "partnering."  It is here that the clinician is no longer in the center of or more important to the relationship.  In partnering/coproduction, the patient and the clinician are equal.  Here they give and take equally.  This is the place where decision-making works best, as both parties respect each other's expertise.

This slide was really great.  It was the results from the CFF Mind of the Community Survey given last year.  It shows that while patients do tend to primarily trust their doctors with regard to CF research/drugs (70%), clinical care (50%) and medical stages of CF (34%), they actually trusted their peers primarily when it came to sticking to a treatment plan (37%), day-to-day life (67%) and CF and relationships (56%).  It's clear that in many important areas people/families with CF trust people/families with CF more than their often-central care team.  For this reason, we must treat CF differently than we have in the past.  We can no longer have a one-way communication relationship, it must be partnership - it must be coproduction.

Alongside Kathy Sabadosa (a CF Mom) and Melanie Abdelnour (a person with CF), Cindy George (CFF's Senior Director of Patient Engagement shared about the partnership experience from the CFF's point of view.  Cindy mentioned that the CFF is putting resources toward improving coproduction, most specifically within the CFF's mental health initiative, with the addition of CF resources and guidelines, by way of the Success with Therapies Research Consortium and through the Quality Improvement Learning Networks such as the CF Learning Network.

I can personally attest that the CFF is putting more resources towards building better partnership with patients and their clinicians in order to improve CF care.  This new focus from the CF Foundation has supported the work I've been a part of with the CF Learning Network (CFLN) for three years now.  In particular, over the last year, I have been working within the CFLN to improve CF Center-level coproduction.

If coproduction is patients and clinicians working together as equals, then "center-level coproduction" is when a group of patients and a group of clinicians work together as a team to improve CF care at the CF Center level (such as improving the wait time in the clinic, beefing up infection control, improving the clinic's communication system, etc).  "Individual-level coproduction" is what we refer to as partnership between a individual clinician and a individual patient at point of care/within the clinic room, which is also very important.  But I have been working more directly in center-level coproduction.

This year, I took the research I have had the privilege to gather through the CFLN and submitted it as a poster which was accepted at NACFC 2018.  It was then accepted as a presentation at the "Quality Improvement Thematic Poster Session."  It was really rewarding to be able to teach others about the good work happening within the CF Learning Network through our participating CF quality improvement teams at 27 CF Programs across the US.

I am really hopeful that next year I can work with more of our patient and family members within the CF Learning Network to submit their own posters.  We need more people with CF and their family members presenting research at NACFC.  I fully believe that, as patients - members of the clinical team, we know a lot about CF and we should be demonstrating that knowledge alongside researchers at NACFC.

7.) There's a company working towards PERT-free food.  

What if you could offer your kid a snack without the need for enzymes?  That's what the gang at GlycosBio are working on.  It's a very interesting concept.

8.) Bowel, bladder and sexual dysfunction is not uncommon in CF.  Better posture and strengthening core and pelvic muscles may help.
Physical therapists are being used more and more CF care.  There are common issues prevalent in CF that might benefit from work with a physical therapist.  For example, bowel and sexual dysfunction is common in CF.  

This is what it looks like for your diaphragm and pelvic floor when you cough.  This is why for many there is loss of bladder and bowel control, the muscles may be trying to do their job but the pressure from coughing can be too much.

Physical therapist Karen Von Berg explained that building good posture and core muscles may help with the stress the muscles often find themselves under due to CF's effect on the body.    

Of course, Karen Von Berg were also reminded us that we should all be sitting at a 35 degree angle when we poop, CF or not. (Credit: Karen Von Berg, D.P.T. The Johns Hopkins Hospital, "Collaborative Approach to GI Management: A Team Perspective")

9.) One way to address DIOS in CF may be massage of the abdomen.  

I don't have a ton of information about this session but I thought it was good.  Karen Von Berg also explained that massage can help DIOS issues.  It's unclear why it works - if it's because massage on the abdomen gets the blood flowing on that area which improves motility or if it's because massaging itself can massage the poop to the right place.  While Karen didn't have a video on how to give massage but she suggested massaging along the bowel: up the ascending bowel over to and down the descending bowel. (Credit: Karen Von Berg, D.P.T. The Johns Hopkins Hospital, "Collaborative Approach to GI Management: A Team Perspective")

 10.) There is still a lot we don't yet know about pulmonary exacerbations.

How do we define a "pulmonary exacerbation?"  Doctors don't agree.  What do we treat it with?  What do we use to treat?  Where should we treat it?  Who should monitor treatment?  When is treatment stopped and when is the pulmonary exacerbation resolved?  How do we classify it as mild, moderate or severe?  How does the patient/family weight in?  These are just some of the questions doctors and researchers are trying to answer.
11.) There is debate within the clinician community on whether IV antibiotics should be given at home vs. in the hospital.
Before I share more, I must outline what the debate between two really great CF doctors was about.  They debated whether research points us to believe IV antibiotics (once started in the hospital and proven to be effective for an individual patient) should be continued at home vs. in the hospital.  

The debate was convincing on both sides.  It seems that the issue is very patient dependent and results, positive or negative, can due to a complexity of things.  Data may tend towards telling us that FEV1 results seem to improve better if you finish a course of IV antibiotics in the hospital.  But we know that FEV1 is just one measure of someone's overall health in CF.  Data also tends towards telling us that quality of life may improve when a course of IV antibiotics are given at home.

What I came away with for my own family was that IV Antibiotics can be given at home but need to include, at minimum, 1-2 weekly labs, weekly clinic visits, PICC line care and good communication with the home care team.

The conclusion of the debate brought about a few important points both doctors agree on. Both doctors agree that the CF community needs to be more aggressive with treatment of exacerbations.  And, if IV antibiotics are to be given at home, there should be shared decision making (partnering between the clinicial team and the patient) and good processes for administering and monitoring.

This debate will likely continue as more research is being done on it.  STOP 3 is a study that might provide some insights as well as ongoing quality improvement projects from clinics. (Credit: Elliot Dasenbrook, M.D. M.H.S. Cleveland Clinic, "Exacerbation Treatment at Home is as Effective as Exacerbation Treatment in the Hospital. Pro")

While I was swayed slightly by the data to stay in the hospital for IV antibiotics as long as possible, I find myself more than frustrated that the the hospital is hugely disruptive to sleep and family life.  I did gain a better appreciation that IV antibiotics given at home may mean people who get IVs at home tend to push themselves too fast to get back in the saddle (they go back to work, school, family life, etc too fast) and may not fully recognize their own need to rest stress-free. (Credit: Don Sanders, M.D. M.S. Indiana University, "Exacerbation Treatment at Home is as Effective as Exacerbation Treatment in the Hospital. Con")

12.) Respiratory virus infections, including RSV and Influenza A, may promote P. aeruginosa biofilm growth which transitions to chronic infection.  
This slide shows the the presence of RSV (which is one of the cold viruses) has a correlation with more PA (bacteria in green).  But, scientists have been trying to figure out why. 

Apparently, when a virus goes in to the epithelial cells (the outside cell of the lung), the epithelial cells create a substance called interferon.  Interferon signals to other cells that an infection is near and helps other cells resist the virus.  But some scientists now hypothesize that interferon stimulates PA to develop a biofilm.  This biofilm is a protective "bunker" that protects the PA from the body's defenses. Possibly this biofilm establishes chronic infection. (Credit: Brian Kocak, University of Pittsburg, "Interferon Signaling Induces Metabolic Shifts In The CF Airway Epithelium To Promote P. Aeruginosa Biofilm Growth")

13.) Microbial ecology (how bacteria, viruses, etc. interact with each other) is important in CF and shouldn't be ignored.
In the session called, "Mucin as a Nutrient Source for Staphylococcus Aureus Invitro & In CF Associated Chronic Rhinosinusitis," it was presented that, in general, pathology (or CF disease) is determined as much by the conditions of the environment inside the lungs as by the presence of a pathogen (bad bacteria/virus, etc).  In other words how sick with CF you are is probably less about whether you have a bad bacteria in your lungs or sinuses but it's more about the interaction of that  bacteria with other bacteria.  (As Brian explained it to me: it's not whether Johnny is a bad kid or a good kid, it's whether Johnny is a bad kid when he gets around the bad kids at school).

As demonstrated on this slide, the healthy lung environment (left side) has plenty of oxygen (O2) and normal food (GLc/glucose).  But when there's a viral infection (such as an infection in the sinuses, also called "rhinosinusitis"), oxygen levels (O2) fall and nitric oxide levels (NO) rise and lactate replaces glucose (GLc).  It is here, in this new environment that bacteria that was harmless suddenly becomes disease-causing.

This session also discussed how chronic rhinosinusitis (an infection in the sinuses) can help promote Staph A. infections (see above).  For this reason and the reason written above, it's becoming more clear that it's not enough to avoid active Staph A. contamination.  We should also be avoiding any respiratory infection at all, even seemingly harmless ones, since chronic diseases are the result of complex microbial interactions.  As Brian explained to me: it takes a village to get a chronic disease, so it's best to avoid the entire village if one can. (Credit: Sarah Lucas, PHD Candidate, University of Minnesota, "Mucin as a Nutrient Source for Staphylococcus Aureus Invitro & In CF Associated Chronic Rhinosinusitis")

14.) Nutrition options in the CF world continues to expand.  
A few years ago, I learned about Kate Farms at NACFC.  We love Kate Farms feeding tube formula at my house and use it nearly every day.  Now, there is another company on the market called "Functional Formularies" which offers organic, whole-food feeding tube formulas and whole meal replacements.

15.)  Physical therapists are being added to many CF teams and they are doing great things with PT.

Bennett has started benefiting from physical therapy.  So, I was very interested to attend this session on physical therapy to learn more about the benefits of physical therapy.  What was funny was when I arrived for the session, I learned it was Bennett's CF physical therapist giving the presentation.  I guess that's when you know you're at the right place when you want to better understand your clinician's advice and find out that it's your clinician who is teaching others - ha! (Credit: Renalli Arcinas, D.P.T., Children's Medical Center Dallas"Putting the Pieces Together: Comprehensive Application of PT Interventions in CF".)
16. There's a new a college scholarship opportunity for siblings of people with CF through Vertex. 
This may not be new news but this was the first time for me to hear that Vertex has a $5,000 scholarship program which includes siblings of people with cystic fibrosis as well as caregivers (yay for Vertex for recognizing CF affects the entire family!).  To apply, click here:

Speaking of Vertex, Brian and had an opportunity to meet Paul Negulescu from Vertex at NACFC.  I asked Paul if he would take a picture with Brian and me as we wanted to show the picture to Bennett when we returned home.  I always tell Bennett when I go to NACFC that "I am going to meet with the doctors and scientists trying to find a cure for CF."  Now that a med that may actually benefit Bennett is likely to be out in the next year or so, I decided to show Bennett the picture of the person who helped create it.  We don't know what the future will hold for Bennett but it feels really great to know there are a lot of people like Paul out there trying to find a cure for cystic fibrosis.

In conclusion, I wanted to provide free online resources for anyone who is interested in reading more about the research provided at the 2018 NACFC.  Click here to learn more:
Plenary 1:
Plenary 2:
Plenary 3:
The list of NACFC 2018 sessions:
The list of NACFC 2018 posters: (see what research is happening in the CF community)
Poster Session Abstracts: (read these!  They are interesting!)
Symposium Summaries:
Author Index: (find out what research your clinician might be involved in these days)
Facebook NACFC QA Sessions: